The increasing complexity of emerging therapies and the need for faster development have led to the adoption of advanced ...

Qure's valuation appears slightly high relative to peers, with limited upside potential. Click here to learn why QURE stock ...

After 44 days, Kendric Cromer, 12, left the hospital. While his family feels fortunate that he was the first to receive a ...

Confounding data from a Pfizer clinical trial have rattled the field of gene therapy for Duchenne muscular dystrophy ...

Emily Pink, 21, has cystic fibrosis, a rare genetic disorder that causes a build-up of sticky mucus in her lungs and ...

Cas9, a technology that has moved from lab to clinic at lightning speed. Scientists have already used gene editing to improve ...

Scientists have discovered that certain changes in the DNA of cells, known as mutations, like the IDH mutation, can act as ...

The first patient has been dosed in an open-label Phase 1/2 clinical trial of AMT-162, uniQure's one-time gene therapy for ...

Alnylam's Yvonne Greenstreet, at #STATSummit, predicts vutrisiran will become multibillion-dollar product for ...

All cancer mutations that cause drug resistance fall into one of four categories. New research has detailed each type, helping to uncover targets for drug development and identify potential effective ...

Researchers identified key gene expression differences in addiction-relevant brain regions that provide new insights into ...

Gene therapy could help obese kids avoid health problems associated with excess weight, including arthritis, a new study ...